Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D

• ClinicalTrials.gov Identifier: NCT03301532
• Recruitment Status: Active, not recruiting
• First Posted: October 4, 2017
• Last Update Posted: December 23, 2022
• Sponsor: University of Texas Southwestern Medical Center
• Collaborator: National Institute of Neurological Disorders and Stroke (NINDS)
• Information provided by (Responsible Party): Juan Pascual, University of Texas Southwestern Medical Center

Tracking Information

• First Submitted Date: August 29, 2017
• First Posted Date: October 4, 2017
• Last Update Posted Date: December 23, 2022
• Actual Study Start Date: June 5, 2018
• Estimated Primary Completion Date: June 18, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 28, 2017)

Ketosis [ Time Frame: 3 years ]

Changes in blood betahydroxybutyric acid level.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: September 28, 2017)

• EEG [ Time Frame: 3 years ]
  Changes in abnormal EEG discharges.
• Seizure rate [ Time Frame: 3 years ]
  Changes in observable seizure numbers.
• Glycemia [ Time Frame: 3 years ]
  Changes in blood glucose levels.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
• Official Title: Compatibility of Triheptanoin (C7) With the Ketogenic Diet in Patients Diagnosed With Glucose Transporter Type 1 Deficiency
• Brief Summary: To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
• Detailed Description: This is a single site, open label proof of principle exploratory trial to investigate the compatibility of C7 oil with the ketogenic diet in subjects diagnosed with G1D. The ketogenic diet will have been previously described by the patient's treating physician independently of this study and for clinical reasons. The ketogenic diet supplies over 50% of calories from fat, subjects, who are already tolerating over this much fat as part of their previously prescribed ketogenic diet, will replace 45% of their daily caloric intake with the triheptanoin for 24 hours, during a 48 hour inpatient stay. Subjects will have a continuous EEG to monitor for any potential C7 related changes in seizure before, during, and after triheptanoin oil ingestion.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: GLUT1DS1

Intervention

Drug: Triheptanoin

Dietary supplementation with triheptanoin

Other Name: C7 oil

Study Arms

Experimental: Patients on a ketogenic diet

This is a one arm study were patients will be receiving an oil called triheptanoin. Patients will be consuming triheptanoin 4 times over the course of one day. The triheptanoin oil will take up 45% of their daily calories on the day the day they are taking the oil.

Intervention: Drug: Triheptanoin

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: December 29, 2021): 10
• Original Estimated Enrollment (submitted: September 28, 2017): 15
• Estimated Study Completion Date: July 5, 2024
• Estimated Primary Completion Date: June 18, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical genotyping at a CLIA-certified laboratory.
• Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have taken place for 2 months). The initiation of a ketogenic diet is previous to - and thus is not part of this study.
• Males and females 30 months to 35 years and 11 months old inclusive.

Exclusion Criteria:

• Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
• Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome, crohn's disease, or colitis that could increase the subject's risk of developing diarrhea or stomach pain.
• Subjects with a BMI (body mass index) greater than or equal to 30.
• Subjects currently not on ketogenic diet.
• Women who are pregnant or breast feeding may not participate. Women who plan to become pregnant during the course of the study, or who are unwilling to use birth control to prevent pregnancy (including abstinence) may not participate. Females age 10 and over will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects will be asked to agree to abstinence or another form of birth control for the duration of the study.
• Allergy/sensitivity to C7
• Previous use of triheptanoin less than 1 month prior to study initiation.
• Treatment with medium chain triglycerides in the last 24 hours.
• Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain disorder (such as Alzheimer's disease) that would confound assessment of cognitive changes, in the opinion of the investigator.
• Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements.
• Inability or unwillingness of subject or legal guardian/representative to give written informed consent, or assent for children age 10-17,
• Addition of a new antiseizure drug in the previous 3 months.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 30 Months to 35 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03301532
• Other Study ID Numbers: STU 102015-091; R01NS094257-01A1 ( U.S. NIH Grant/Contract )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Juan Pascual, University of Texas Southwestern Medical Center
• Original Responsible Party: University of Texas Southwestern Medical Center
• Current Study Sponsor: University of Texas Southwestern Medical Center
• Original Study Sponsor: Same as current
• Collaborators: National Institute of Neurological Disorders and Stroke (NINDS)

Investigators

• Principal Investigator: Juan Pascual, M.D.; Study Principal Investigator

• PRS Account: University of Texas Southwestern Medical Center
• Verification Date: December 2022